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Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
- Published in:
- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-25697-y
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- Article
Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.
- Published in:
- Human Molecular Genetics, 2017, v. 26, n. 8, p. 1535, doi. 10.1093/hmg/ddx058
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- Publication type:
- Article